Research programme: gene editing therapeutics - CRISPR Therapeutics

Drug Profile

Research programme: gene editing therapeutics - CRISPR Therapeutics

Alternative Names: CRISPR-Cas9 gene therapies

Latest Information Update: 24 Jun 2017

Price : $50

At a glance

  • Originator CRISPR Therapeutics
  • Developer CRISPR Therapeutics; Genethon; University of Florida; Vertex Pharmaceuticals
  • Class Gene therapies
  • Mechanism of Action Gene modulators; Immunomodulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Beta-thalassaemia; Cancer; Haemoglobinopathies; Mucopolysaccharidosis I; Sickle cell anaemia
  • Research Amyotrophic lateral sclerosis; Cystic fibrosis; Dementia; Duchenne muscular dystrophy; Glycogen storage disease type I; Unspecified

Most Recent Events

  • 19 Jun 2017 CRISPR Therapeutics has patent protection for CRISPR-Cas9 genome editing technology
  • 16 May 2017 CRISPR Therapeutics collaborates with the University of Florida for gene editing therapeutics for Amyotrophic lateral sclerosis and Dementia
  • 16 May 2017 Early research in Amyotrophic lateral sclerosis in USA (unspecified route)
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